Veritas In Silico developed technologies in informatics for identifying structural motifs on mRNA that can be drug targets, and in experimental biology for verifying the structures of target motifs as well as for screening. These technologies allow for the discovery of small molecule and antisense oligonucleotide (ASO) drugs targeting mRNA.
Veritas In Silico is engaged in the creation of mRNA-targeted drugs as its core business, using the aibVIS platform, which integrates our specialized AI technologies, including in silico RNA structure analysis, with experimental biology. Through collaborative drug discovery research with pharmaceutical companies, we are working to create mRNA-targeted small molecule drugs, while also developing nucleic acid medicines through our proprietary pipelines.
To deliver treatments for diseases that have been difficult to address with conventional drug discovery approaches, we will continue to open up new frontiers in the pharmaceutical market through the innovative use of our proprietary AI drug discovery platform, aibVIS.
mRNA is the “blueprint” for the genetic information of proteins produced in the human body. In recent years, as the depletion of druggable target proteins has become a concern in small molecule drug discovery, the ability to regulate mRNA would make it possible to treat a wide range of diseases that have been difficult to address with conventional drug discovery, in much the same way that drug products directly control the function of target proteins. It is therefore expected to help meet unmet medical needs and address the depletion of drug targets. However, because mRNA does not adopt a single fixed structure and it is difficult to perform precise structural analysis at the start of drug discovery research, there had been an industry-wide recognition that it is difficult to carry out small molecule drug discovery targeting mRNA.
To address these challenges in mRNA drug discovery, we have developed and evolved our proprietary AI drug discovery platform, aibVIS. By integrating in silico RNA structure analysis with experimental technologies and combining multiple rule-based AI and data-driven AI approaches, aibVIS enables efficient and reliable mRNA-targeted drug discovery.
The key strength of aibVIS is its ability to rapidly and comprehensively search for and identify the “keyhole”-like structural motifs on mRNA that serve as target structures, through in silico RNA structure analysis based on statistical mechanics and thermodynamic theory. This has made it possible to overcome the characteristics of mRNA that have made it difficult to target in drug discovery, and to identify stable structures to which small molecule drugs can bind.
aibVIS provides the following drug discovery process in a one-stop manner.
1. Target search
We obtain mRNA sequence data from the NCBI database and use our proprietary RNA structure analysis software, MobyDick®, to identify target structures suitable for small molecule drugs. This target search achieves a success rate of approximately 98%.
2. Screening
Using qFRET, an improved form of FRET (fluorescence resonance energy transfer), we rapidly and sensitively identify hit compounds that bind to the target structure from tens of thousands to hundreds of thousands of compounds. The screening success rate is also very high at approximately 96%, and further efficiency gains are expected through the introduction of our data-driven AI, AISLAR.
3. Hit to lead
We confirm the validity of hit compounds through cell-based experiments and verify their effects in cells. Using analytical technologies such as BLI, ITC, and NMR, we analyze binding strength and characteristics in detail and select lead compounds for the next stage.
4. Lead optimization
We determine the three-dimensional structure of the complex formed by the target structure and the compound using NMR and X-ray crystallography, and optimize the activity and physicochemical properties of drug candidate compounds using quantum chemical calculations. We also predict side effects using our in-house rule-based AI, search4loop.
aibVIS has a clear competitive advantage in efficiently identifying druggable structural motifs on mRNA and defining target structures. This technology is patented in Japan, Europe, and the United States as a “method for screening compounds that regulate RNA function,” and the confidentiality of our in-house developed AI software further ensures the exclusivity of the entire platform.
We operate both a “platform-based” business that provides technology to pharmaceutical companies and a “pipeline-based” business that creates proprietary pipelines (drug candidates).
1. Research activities in our platform business
In our platform-based business, we are advancing the creation of mRNA-targeted small molecule drugs through collaborative drug discovery research with multiple pharmaceutical companies. Small molecule drugs can be administered orally as tablets and also have the advantage of relatively low manufacturing costs, which makes it possible to provide treatment widely to many patients. Currently, our most advanced project is in the “lead compound creation” stage.
2. Research activities in our pipeline business
In our proprietary pipeline business, we are actively engaged in research and development of nucleic acid medicines (ASOs). With rare diseases as our primary target, we have the strength of being able to obtain drug candidate compounds in as little as eight months. Currently, as our first pipeline, we are developing an ASO targeting the p53 gene for the prevention of acute renal failure after cardiovascular surgery, and we filed a substance patent in December 2025.
We are also advancing ASO projects targeting amyotrophic lateral sclerosis (ALS) and primary graft dysfunction after lung transplantation.
Another key highlight of our future proprietary pipeline development is our proprietary drug delivery system, Perfusio. Using a single set of catheters, this system directly administers and recovers drugs to and from the target organ, with the potential to enhance therapeutic efficacy, reduce side effects, and significantly shorten both the duration and cost of clinical trials.
About drug delivery system
We are advancing our research and development through a two-site structure Kawasaki Research Institute for core technologies and platform research, and Niigata Research Institute for cell-based evaluation and pipeline research. We are also actively strengthening our technological competitiveness through external collaboration, including joint research with academia and the co-development of high-speed, high-precision RNA structure measurement technology with Dexerials Corporation.
Our medium-term management goal is to establish ourselves as a specialty pharmaceutical company by 2030. The application range of mRNA drug discovery technology is broad, and as future business diversification, we are also considering applications in the agricultural chemicals field and licensing out the Perfusio system to other companies.
We will continue our challenge to create new mRNA-targeted medicines through the power of AI drug discovery and bring hope to patients suffering from diseases for which there are still no treatments.